Dyno Therapeutics’ Post

So far our #immune_system is the main challenge in translating a lab experiment of #gene_therapy into reality. Gene therapy holds immense potential for treating a vast array of diseases, but its greatest challenge lies in the immune system and delivery mechanism. Since its inception, effectively transporting a gene to the targeted cells has remained a formidable obstacle. While #Adeno_Associated_Viruses (AAVs) have shown promise, our body's immune system develops antibodies against them, rendering subsequent doses ineffective. This "one-shot" limitation significantly hinders their utility. Furthermore, the long-term efficacy of gene therapy is yet to be definitively established. There's a concern that the therapeutic effects might diminish over time as cells undergo #mitosis (cell division) and create copies with potentially diluted or even absent genetic modifications. These challenges necessitate further research in developing more robust and targeted delivery methods. Overcoming these hurdles will unlock the true potential of gene therapy, paving the way for a new era of medicine. Researchers are shifting their focus towards alternative delivery mechanisms for gene therapy. These mechanisms include exosomes and mRNA-coded lipoproteins. This shift is likely due to the limitations of current methods. By doing that they may be able to dodge immune system but these delivery vehicles are not that effective when compared to #viral_vectors. Heidi Ledford have highlighted some of these challenges and future hope in his note. https://lnkd.in/dphHuv7n

🧬 The FDA approved 2 gene edited therapies in December last year! The FDA approved Casgevy and Lyfgenia, two groundbreaking gene therapy treatments for sickle cell disease. Developed by Vertex Pharmaceuticals and Bluebird Bio, respectively, these therapies mark a pivotal moment in medical history! Casgevy, the first FDA-approved CRISPR gene editing therapy, utilizes DNA code to repair damaged cells. Lyfgenia uses cells from the patient’s body to potentially decrease or stop vaso-occlusive events in one treatment. These therapies can replace traditional treatments, including riskier treatment options including bone marrow transplants, by precisely editing faulty genes in stem cells, which could potentially save lives. At QHP Capital, we believe that new gene editing approaches will drive future advancements and treatments in this area. Watch the video below for your industry check-up: #lifesciences #privateequity #celltherapy #geneediting

📢 𝗘𝘅𝗰𝗶𝘁𝗶𝗻𝗴 𝗕𝗿𝗲𝗮𝗸𝘁𝗵𝗿𝗼𝘂𝗴𝗵 𝗶𝗻 𝗚𝗲𝗻𝗲 𝗧𝗵𝗲𝗿𝗮𝗽𝘆! Exciting news emerges from the realm of gene therapy! Peter Coleman, a key figure at RoslinCT, foresees a ground-breaking era in treatments. He emphasizes, "These will be the treatments of the future. They are not just tackling the symptoms; they are providing cures for individuals by genetically modifying the cells in people's bodies." Rowan Flynn, Principal Scientist at RoslinCT, sheds light on the versatility of Crispr technology, stating, "While Casgevy is specific for beta thalassaemia and sickle cell disease, the Crispr technology as a whole is extremely versatile and can be widely applied to other diseases that have some sort of genetic component to them." This marks a pivotal moment for gene therapy, hailed for its potential to provide cures rather than just symptom management. Although much is yet to be unveiled about this particular treatment, its transformative impact on healthcare is undeniable. Pricing remains uncertain, with no set cost for Casgevy in the UK. According to Vertex Pharmaceuticals the US list price stands at $2.2 million. As discussions with health authorities progress, the focus is on securing reimbursement and swift access for eligible patients. While assessors will determine cost-effectiveness, the development kindles hope for a brighter, healthier future. #GeneTherapy #MedicalInnovation #HealthcareBreakthrough #Casgevy #ResearchAdvancement Find out more here: https://hubs.la/Q02fm-tl0

Gene therapies rely on modifying cells outside the body. We lacked the technology to deliver the therapy to specific cells. Today, a novel delivery method can deliver the therapy inside the body. Jennifer Hamilton and her team from the University of California, Berkeley, developed a new, precision-targeted delivery method for CRISPR-cas9. How does it work? 1. Delivery vehicle The method consists in using a carrier membrane, called enveloped delivery vehicles (EDV). These vehicles are highly modified versions of the HIV-1 virus envelope. 2. Precision delivery These EVDs were modified to express two antibodies at their surface. These antibodies act as a homing mechanism, targeting specific cell types. 3. In vivo delivery The method was used to target T cells in live mice and turn their cells into anti-CD19 CAR-T cells. The EVD delivered the CD19 transgene and knocked out native T cell receptors. There was no uptake by bystander cells, including liver cells. This approach skips the need to engineer cells outside the body entirely. The manufacturing process is streamlined. Gene therapies are more accessible to patients. PS: What do you think of in vivo gene therapy? Let's chat 😊 #GeneTherapy #CRISPR #CGT

I know I normally post my own summaries, but I really like this one from Tasmin Lacourte. So, here it is. No notes. #cgt #crispr #viralvectors (ish) #targetedtherapy #solidtumors

With 12 gene therapies (GT) now FDA-approved for rare disease, spanning three different technologies, how important is the technology itself in guiding the commercial success of a GT? Read my piece exploring this question, using beta-thalassemia as a case study following the recent approval of Vertex/CRISPR Therapeutics CASGEVY - the first CRISPR GT to be approved in the US/EU - and share your thoughts!

CasRx system: a new method for lncRNA functional screening. The CasRx system utilizes the Cas13d enzyme of the CRISPR-Cas family and has become a revolutionary tool in long non-coding RNA (lncRNA) research, providing a new method for functional screening of lncRNA. Unlike traditional CRISPR-Cas9 technology that targets DNA, CasRx operates at the RNA level, allowing precise silencing or editing of lncRNAs via specific guide RNA (gRNA) sequences. The method is highly specific, simplifies operation, does not require cell repair mechanisms, and is versatile across a variety of cell types and tissues. The introduction of CasRx paves the way for breakthrough advances in functional genomics, providing new ways to understand the role of lncRNAs in disease mechanisms and potential therapeutic applications. By enabling targeted lncRNA intervention, CasRx is expected to develop innovative therapeutic strategies and gene therapy applications, marking a major advance in the exploration of gene regulation and disease correction.

Excited to share our latest research!🔬 We've developed a gene editing strategy for blood stem cells that activates the expression of a therapeutic protein solely upon their differentiation into myeloid cells, therefore preserving stem cells' innate functions. This technique enables systemic distribution of a therapeutic protein, even across the blood-brain barrier. We're very excited about the possibilities this technology unlocks for more targeted, effective, and safer gene therapies.   Read the full paper here: https://lnkd.in/evUe5Vr2   #GeneTherapy #StemCellEditing #BiomedicalResearch #MyeloidLineage #InnovativeTherapeutics

CasRx system: a new method for lncRNA functional screening. The CasRx system utilizes the Cas13d enzyme of the CRISPR-Cas family and has become a revolutionary tool in long non-coding RNA (lncRNA) research, providing a new method for functional screening of lncRNA. Unlike traditional CRISPR-Cas9 technology that targets DNA, CasRx operates at the RNA level, allowing precise silencing or editing of lncRNAs via specific guide RNA (gRNA) sequences. The method is highly specific, simplifies operation, does not require cell repair mechanisms, and is versatile across a variety of cell types and tissues. The introduction of CasRx paves the way for breakthrough advances in functional genomics, providing new ways to understand the role of lncRNAs in disease mechanisms and potential therapeutic applications. By enabling targeted lncRNA intervention, CasRx is expected to develop innovative therapeutic strategies and gene therapy applications, marking a major advance in the exploration of gene regulation and disease correction. (https://lnkd.in/edisgpwq).

Capsida’s David Goertsen, Ph.D., will be presenting at the 4th Next Generation Gene Therapy Vectors Summit, focused on Innovating Gene Therapy Capsid & Payload Design. Dr. Goertsen’s presentation will review Capsida’s progress in AAV capsid engineering to develop gene therapy candidates with improved safety and efficacy profiles for PD-GBA and genetic epilepsy due to STXBP1:   • Platform advancements leading to breakthrough, IV-delivered engineered AAV capsids for the CNS • Generations of directed evolution yielding highly targeted AAV capsids with increased therapeutic index • Potential for Capsida's engineered AAVs to treat neurological disorders across all age groups   This summit brings together more than 60 biopharma leaders, service providers, and academic pioneers to discuss the latest advancements and innovations in vector development for gene therapies.    📅 Event: 4th Next Generation Gene Therapy Vectors Summit  📍 Location: Boston  🗓️ Date: June 14 at 2:15pm   #GeneTherapy #AAV #Biopharma #Capsida #Innovation #NextGenVectorsSummit #NeurologicalDisorders #R&D